HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP
This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.
Eligibility
Sex
ALL
Min age
12 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* Participants with diagnosis of EPP who are participating (or who have participated) in a prior Disc Medicine bitopertin study and who have completed the randomized treatment phase and End-of-Study visit
* Aged ≥12 years upon study consent
* Body weight ≥32 kg for participants \<18 years of age and BMI ≥18.5 kg/m2 for adult participants
* Willing to practice highly effective methods of birth control (both males who have partners of childbearing potential and females of childbearing potential during the study, while taking study drug, and for at least 30 days after the last dose of study drug.
* Negative urine or serum pregnancy test (females of childbearing potential).
* Able to understand the study aims, procedures, and requirements, and provide written informed consent (and assent if necessary).
* Able to comply with all study procedures.
Exclusion Criteria:
* Participants who have an ongoing SAE from a clinical study that is assessed by the investigator as related to bitopertin
* Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgement of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude participation in the study
* Condition or concomitant medication that would confound the ability to interpret clinical, clinical laboratory, or participant diary data, including a major psychiatric condition that has had an exacerbation or required hospitalization in the last 6 months
* Planned treatment with afamelanotide or dersimelagon during the study
* Planned use of any drugs or herbal remedies known to be strong inhibitors or inducers of cytochrome p450 (CYP)3A4 enzymes throughout the study
* If female, pregnant, or breastfeeding
* Participation in any other clinical protocol or investigational trial, other than Disc Medicine bitopertin trials, that involves administration of experimental therapy and/or therapeutic devices within 30 days of Day 1
* Score of PHQ-8 ≥10 at screening or imminent suicidal risk identified by the C-SSRS as defined as suicidal ideation with intent (Grade 4 or 5) within the last year or any suicidal behavior within the last 5 years.
* Consumption of grapefruit/Seville orange and products containing these for 14 days prior to first dose of study drug and throughout the study
Primary outcome measure(s)
Incidence of treatment-emergent adverse events — up to 5 Years
Incidence of clinically abnormal vital signs — up to 5 Years
Incidence of clinically abnormal physical exam — up to 5 Years
Incidence of abnormal laboratory test results — up to 5 Years
Assessment of Patient Health Questionnaire (PHQ-8) — up to 5 Years The Patient Health Questionnaire (PHQ-8), an 8-item participant-report measure for screening for depression and for establishing depression severity. The total score ranges from 0-24, with a higher score indicating greater depression symptom severity.
Assessment of C-SSRS — up to 5 Years The C-SSRS is a clinician-rated scale that assesses suicidality from ideation to behaviors and monitors the potential emergence of suicidality in clinical studies.
Trial sites (26)
Facility
City
Region
Status
University of Alabama Hospital
Birmingham
Alabama
University of California San Francisco
San Francisco
California
University of Miami Miller School of Medicine
Miami
Florida
Massachusetts General Hospital
Boston
Massachusetts
MetroBoston Clinical Partners
Boston
Massachusetts
Mount Sinai Hospital
New York
New York
Atrium Health Wake Forest Baptist
Winston-Salem
North Carolina
Remington-Davis Clinical Research
Columbus
Ohio
Einstein Medical Center
Philadelphia
Pennsylvania
University of Texas
Galveston
Texas
Fred Hutchinson Cancer Center
Seattle
Washington
Royal Prince Alfred Hospital
Camperdown
New South Wales
The Royal Melbourne Hospital
Parkville
Victoria
UZ Leuven
Leuven
Belgium
CHU de Nantes - Hôtel Dieu, Service de dermatologie
Nantes
France
Centre d'Investigation Clinique (CIC) Hôpital Bichat - Claude-Bernard
Paris
France
Charité - Universitätsmedizin Berlin, Institute of Allergology
Berlin
Germany
Klinikum Chemnitz gGmbH
Chemnitz
Saxony
Children's Health Ireland (CHI)
Dublin
Ireland
Instituto Dermatologico San Gallicano Istituti Fisioterapici Ospitalieri IRCCS
Roma
Italy
Erasmus MC
Rotterdam
Netherlands
Hospital Clinic de Barcelona
Barcelona
Spain
Karolinska University Hospital
Stockholm
Sweden
Guy's and St Thomas' NHS Foundation Trust
London
England
Clinical Research Centre, Ninewells Hospital & Medical School , NHS Tayside
Dundee
Scotland
Photobiology Unit, Salford Royal Hospital
Salford
United Kingdom
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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